Treatment Options

Our greatest goal for you/your child with epidermolysis bullosa (EB) is to ease suffering and provide a chance at a full life, and we have proven that this can be achievable.

Until recently, treatments for epidermolysis bullosa did not exist beyond providing daily care to manage wounds and blisters. That has now changed. We are proud to have recently developed two groundbreaking, potentially life-changing FDA-approved EB treatments here at Stanford, one of which is only offered at a few centers worldwide, including ours.

• Our treatments (skin graft therapy and gene therapy topical gel) work by changing your/your child’s skin genes, offering a chance at healing chronic wounds and truly lessening pain and suffering. Some of our treatments can be combined for even more potential healing.
• To create a smooth care journey, we provide our patients with a dedicated clinic coordinator who helps you arrange care visits, ensures that your multispecialty care team is always updated, offers you helpful referrals, and manages international referrals.

We provide all EB treatments available, including the following:

Therapy via genetically corrected, autologous skin grafts (ZEVASKYN® pz-cel therapy)

We are proud to have pioneered this revolutionary treatment at Stanford for chronic, nonhealing wounds for people with severe recessive dystrophic epidermolysis bullosa (RDEB). Skin graft therapy offers the first chance at healing wounds that have been present for years.

People with RDEB have gene differences that cause their skin to be highly fragile. Sometimes, they may develop more complex symptoms, such as mitten-deformity (when fingers and toes fuse together), skin cancer, and organ damage. Healing chronic wounds helps lessen the chance of these conditions.

With skin graft therapy, we have the laboratory grow sheets of new, stronger skin using your/your child’s own cells and tissue (autologous). In the lab, these sheets of skin are reinforced with collagen. This treatment can make your/your child’s skin stronger—able to resist tearing, breaking, and blistering—and less painful.

Study outcomes for skin graft therapy

At Stanford Medicine, we have been researching epidermolysis bullosa for over 30 years to discover new treatments that help to provide long-term relief of painful skin wounds. In the early 2000s, our doctor-researchers developed a way to change a person’s skin cells with genetics, which resulted in our topical gel and skin graft treatments. We are proud to have been one of two hospitals in the world to participate in a phase III clinical trial of the skin graft therapy, which led to its FDA approval and a commercial drug that is now available. Study outcomes showed that 81% of treated wounds were at least half healed six months after the pz-cel treatment, and none of the treated areas have developed skin cancer (followed through the first year).

Study participants experienced less pain and improved quality of life. They reported being able to attend school and work and to perform daily activities with more ease, and expressed hope for a better future.

What to expect with skin graft therapy

• We begin by taking two small biopsies (removing small pieces of your/your child’s skin) to obtain cells.
• Next, we send the biopsies to the laboratory, where skin grafts are created by inserting normal collagen VII genes into your/your child’s cells. Once the skin grafts have grown and are ready, we surgically graft them onto your/your child’s large, nonhealing wounds in the operating room.
• The hospital stay is approximately five to 10 days to ensure proper recovery and to help new skin grow.
• Because the skin grafts are derived from your/your child’s own skin cells, the immune system is less likely to reject them (react to them as if they are foreign and attack them).
• Up to 12 pieces of engineered skin grafts, or sheets, can be applied during each operation, and each treats an area about the size of a dollar bill. By closing several open wounds at once, the treatment can provide significant pain relief.

Gene therapy topical gel (B-VEC/Vyjuvek)

Prior to developing the skin graft therapy, Stanford Medicine created the first topical gene therapy gel for severe epidermolysis bullosa after years of research. In 2023, Lucile Packard Children’s Hospital Stanford was one of the few hospitals in the nation to conduct clinical trials leading to its FDA approval. Clinical trials showed complete wound healing in 50% of wounds treated over three to six months. The gel is often used to treat smaller wounds. As wounds and blisters heal, the treatment helps relieve your/your child’s pain.

What to expect with the gene therapy topical gel

• The gel delivers missing normal collagen VII genes into your/your child’s chronic wounds using a viral vector (a modified virus stripped of its disease). The introduced collagen VII protein helps make the skin stronger and more resilient.
• The genetically modified gel can be used on infants 6 months of age or older with dystrophic epidermolysis bullosa (DEB), a serious form of EB. When provided to children during early childhood, the gel may help prevent the development of larger wounds.
• The gel is applied during regular bandage changes each week to help prevent and heal wounds. It is now mostly used as an at-home therapy.

Botanical extract topical gel (Filsuvez®)

We offer another topical treatment derived from birch bark (birch bark triterpenes) that has also received recent FDA approval for treating skin wounds in people with two subtypes of EB: junctional epidermolysis bullosa (JEB)—where gene mutations weaken the connection between the two main layers of skin—and DEB. This treatment likely works by lowering inflammation (swelling, pain, itch) in the skin, which promotes skin renewal and wound healing.

What to expect with the botanical extract topical gel

• The gel delivers birch triterpene to support the skin’s natural ability to repair itself.
• The gel can be used on infants 6 months of age or older.
• A single, thick layer of gel is placed on wounds, via a single daily use tube, then covered with a protective nonadherent dressing.

Whole-person care for severe epidermolysis bullosa

At Stanford Medicine Children’s Health, we provide whole-person care. This means that you/your child will be cared for by a team of many specialists. Together, as one team united by a single care plan, we support you/your child and your family with all of your needs. This includes supporting you as you manage care at home, such as providing training on how to wrap a bandage around an open wound, prevent skin infections, and ease pain and itching so that you/your child can achieve the best chance at a good quality of life.

Your/your child’s care team is personalized to meet specific needs according to your/your child’s type and severity of epidermolysis bullosa. Teams will include a few or all of the following doctors:

• Dermatologists, skin doctors who specialize in EB, will provide treatments and manage your/your child’s care.
• Geneticists and genetic counselors help diagnose the type of EB and help young adults with family planning.
• Wound care specialized nurses train you on EB wound care, bandaging, and how to control and avoid infections.
• Surgeons (plastic), ophthalmologists, otolaryngologists (ENT), and gastroenterologists help care for organs that are affected by EB.
• Oral/maxillofacial and dental specialists provide treatment for dental issues and mouth diseases—complications that can come with severe epidermolysis bullosa.
• Nutritionists/dietitians help you/your child achieve the calories needed to thrive, and help solve difficulty with swallowing and problems with growth.
• Pain and palliative care specialists help relieve and control acute (sudden) and chronic (ongoing) pain.
• Physical and occupational therapists improve your/your child’s movement, sometimes with devices, such as splints and walkers.
• Psychologists/psychiatrists and social workers support you/your child in coping with EB by providing psychosocial support, resolving school and work challenges, and offering support for caregivers.
• Pharmacy and clinical trials coordinators help you manage complex medications and clinical trials.